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    Summary
    EudraCT Number:2017-002213-60
    Sponsor's Protocol Code Number:GX1001
    National Competent Authority:UK - MHRA
    Clinical Trial Type:EEA CTA
    Trial Status:GB - no longer in EU/EEA
    Date on which this record was first entered in the EudraCT database:2018-12-11
    Trial results
    Index
    A. PROTOCOL INFORMATION
    B. SPONSOR INFORMATION
    C. APPLICANT IDENTIFICATION
    D. IMP IDENTIFICATION
    D.8 INFORMATION ON PLACEBO
    E. GENERAL INFORMATION ON THE TRIAL
    F. POPULATION OF TRIAL SUBJECTS
    G. INVESTIGATOR NETWORKS TO BE INVOLVED IN THE TRIAL
    N. REVIEW BY THE COMPETENT AUTHORITY OR ETHICS COMMITTEE IN THE COUNTRY CONCERNED
    P. END OF TRIAL
    Expand All   Collapse All
    A. Protocol Information
    A.1Member State ConcernedUK - MHRA
    A.2EudraCT number2017-002213-60
    A.3Full title of the trial
    A randomized, controlled, open-label, single-ascending dose, phase I/II study to investigate the safety and tolerability, and efficacy of intravenous SGT-001 in male adolescents and children with Duchenne muscular dystrophy
    A.3.1Title of the trial for lay people, in easily understood, i.e. non-technical, language
    A study to investigate the safety, tolerability, and efficacy of SGT-001 in male adolescents and children with Duchenne muscular dystrophy
    A.4.1Sponsor's protocol code numberGX1001
    A.5.2US NCT (ClinicalTrials.gov registry) numberNCT03368742
    A.7Trial is part of a Paediatric Investigation Plan No
    A.8EMA Decision number of Paediatric Investigation Plan
    B. Sponsor Information
    B.Sponsor: 1
    B.1.1Name of SponsorSolid Biosciences Inc.
    B.1.3.4CountryUnited States
    B.3.1 and B.3.2Status of the sponsorCommercial
    B.4 Source(s) of Monetary or Material Support for the clinical trial:
    B.4.1Name of organisation providing supportSolid Biosciences Inc.
    B.4.2CountryUnited States
    B.5 Contact point designated by the sponsor for further information on the trial
    B.5.1Name of organisationSolid Biosciences Inc.
    B.5.2Functional name of contact pointClinical Operations
    B.5.3 Address:
    B.5.3.1Street Address141 Portland Street, Fifth Floor
    B.5.3.2Town/ cityCambridge, MA
    B.5.3.3Post code02139
    B.5.3.4CountryUnited States
    B.5.4Telephone number1617337-4680
    B.5.6E-mailSGT-001_clinicaltrialinfo@solidbio.com
    D. IMP Identification
    D.IMP: 1
    D.1.2 and D.1.3IMP RoleTest
    D.2 Status of the IMP to be used in the clinical trial
    D.2.1IMP to be used in the trial has a marketing authorisation No
    D.2.5The IMP has been designated in this indication as an orphan drug in the Community Yes
    D.2.5.1Orphan drug designation numberEU/3/16/1759
    D.3 Description of the IMP
    D.3.1Product nameSGT-001
    D.3.2Product code rAAV9-CK8-h-μD5
    D.3.4Pharmaceutical form Infusion
    D.3.4.1Specific paediatric formulation No
    D.3.7Routes of administration for this IMPIntravenous use
    D.3.8 to D.3.10 IMP Identification Details (Active Substances)
    D.3.8INN - Proposed INNSGT-001
    D.3.9.1CAS number 2220231-04-7
    D.3.9.2Current sponsor codeSGT-001
    D.3.9.3Other descriptive namerAAV9-CK8-h-μD5
    D.3.10 Strength
    D.3.10.1Concentration unit Other
    D.3.10.2Concentration typenot less then
    D.3.10.3Concentration number6.5E12 vg/mL
    D.3.11 The IMP contains an:
    D.3.11.1Active substance of chemical origin No
    D.3.11.2Active substance of biological/ biotechnological origin (other than Advanced Therapy IMP (ATIMP) No
    The IMP is a:
    D.3.11.3Advanced Therapy IMP (ATIMP) Yes
    D.3.11.3.1Somatic cell therapy medicinal product No
    D.3.11.3.2Gene therapy medical product Yes
    D.3.11.3.3Tissue Engineered Product No
    D.3.11.3.4Combination ATIMP (i.e. one involving a medical device) No
    D.3.11.3.5Committee on Advanced therapies (CAT) has issued a classification for this product No
    D.3.11.4Combination product that includes a device, but does not involve an Advanced Therapy No
    D.3.11.5Radiopharmaceutical medicinal product No
    D.3.11.6Immunological medicinal product (such as vaccine, allergen, immune serum) No
    D.3.11.7Plasma derived medicinal product No
    D.3.11.8Extractive medicinal product No
    D.3.11.9Recombinant medicinal product Yes
    D.3.11.10Medicinal product containing genetically modified organisms Yes
    D.3.11.11Herbal medicinal product No
    D.3.11.12Homeopathic medicinal product No
    D.3.11.13Another type of medicinal product No
    D.8 Information on Placebo
    E. General Information on the Trial
    E.1 Medical condition or disease under investigation
    E.1.1Medical condition(s) being investigated
    Duchenne muscular dystrophy
    E.1.1.1Medical condition in easily understood language
    Duchenne muscular dystrophy
    E.1.1.2Therapeutic area Diseases [C] - Musculoskeletal Diseases [C05]
    MedDRA Classification
    E.1.2 Medical condition or disease under investigation
    E.1.2Version 20.1
    E.1.2Level PT
    E.1.2Classification code 10052655
    E.1.2Term Duchenne muscular dystrophy gene carrier
    E.1.2System Organ Class 10010331 - Congenital, familial and genetic disorders
    E.1.2 Medical condition or disease under investigation
    E.1.2Version 20.0
    E.1.2Level PT
    E.1.2Classification code 10013801
    E.1.2Term Duchenne muscular dystrophy
    E.1.2System Organ Class 10010331 - Congenital, familial and genetic disorders
    E.1.3Condition being studied is a rare disease Yes
    E.2 Objective of the trial
    E.2.1Main objective of the trial
    The primary safety objective of this study is to investigate the safety and tolerability of ascending doses of SGT-001 administered as a single dose IV in adolescents and children with Duchenne muscular dystrophy (DMD).
    The primary efficacy objective of this study is to investigate the efficacy of SGT-001 IV in adolescents and children with DMD, by quantifying expression of microdystrophin in muscle biopsies using western blot (WB).
    E.2.2Secondary objectives of the trial
    • Quantifying expression of microdystrophin via mass spectrometry and immunofluorescence (IF) in muscle biopsies;
    • Measuring skeletal muscle mass and composition utilizing magnetic resonance imaging (MRI);
    • Assessing skeletal muscle-related function and strength;
    • Assessing cardiac and respiratory function;
    • Quantifying change in naturalistic motor function at home as measured by Echo5D® (ambient measurement system device);
    • Assessing daily activity as measured by Actimyo® (wearable accelerometer); and
    • Assessing changes in quality of life and self-reported outcome measures.

    Other objectives include quantifying the expression of serum and muscle biomarkers that is associated with microdystrophin production and/or function.
    E.2.3Trial contains a sub-study No
    E.3Principal inclusion criteria
    1. Male aged 4 years to 17 years, inclusive, at randomization;
    2. Established clinical diagnosis of DMD and documented dystrophin gene mutation predictive of DMD phenotype:
    o For children (aged 4 years to 11 years, inclusive), confirmed absence of dystrophin as determined by muscle biopsy IF
    (≤ 10% dystrophin-positive fibers) or equivalent methodology, performed following randomization and prior to SGT-001 administration;
    3. Anti-AAV9 total/circulating antibody titer <50,000 mU/mL, and anti-AAV9 neutralizing antibody titer ≤1:5;
    4. Stable cardiac and pulmonary function;
    5. For adolescents (aged 12 years to 17 years, inclusive) the following 2 criteria must be met:
    o Unable to walk 10 meters without assistance, AND
    o Score ≤4 on Brooke scale for upper extremity;
    6. For children (aged 4 years to 11 years, inclusive) the following 2 criteria must be met:
    o Walk and climb stairs with/without aid of railing (Vignos scale score
    ≤3), AND
    o Rise from the floor from supine (Gowers) time ≤7 seconds;
    7. A stable dose of at least 0.5 mg/kg/day of oral daily prednisone or equivalent for at least 24 weeks prior to dosing and expected to remain constant throughout the study on a per kg basis, except for protocol-specified dose changes;
    8. Use of medications frequently prescribed for subjects with DMD will be allowed provided the subject has been on a stable dose for 12 weeks prior to Screening and the dose remains constant throughout the study except for adjustments for weight;
    9. Able to understand and comply with all study procedures and have a parent(s) or legal guardian(s) who is able to understand and comply with the study procedure requirements;
    10. Be willing to provide informed assent and have a parent(s) or legal guardian(s) who is willing to provide written informed consent for the subject to participate in the study; and
    11. Subjects of reproductive potential should utilize double barrier contraceptive methods during the duration of the study. These may include (but are not limited to) condom plus other highly effective contraceptive method (such as hormonal contraceptive or intrauterine device).
    E.4Principal exclusion criteria
    1. Any prior or ongoing medical condition, medical history, physical findings, ECG findings, or laboratory abnormality that, in the Investigator's opinion, could adversely affect the safety of the subject, makes it unlikely that the course of treatment or follow-up would be completed, or could impair the assessment of study results;
    2. Abnormal liver function (gamma-glutamyl transferase >1.5 x upper limit of normal [ULN] or total bilirubin >ULN);
    3. Abnormal renal function (cystatin C >1.2 x ULN);
    4. Clinically significant abnormalities of coagulation including international normalized ratio (INR) or activated partial thromboplastin time (aPTT) >1.2 x ULN or platelets <125,000 cell/mm3;
    5. Impaired cardiovascular function (shortening fraction <28%, EF <45% on cardiac MRI or ECHO, according to feasibility/age group);
    6. Respiratory function predictive of (or requiring) the use of daytime ventilatory support, or
    o FVC % predicted <60% (age 4-11 years);
    o FVC % predicted <40%, and/or FVC <1 L (age ≥ 12 years);
    7. Major surgery within 3 months prior to recruitment or planned orthopedic surgery for any time during this study which would interfere with the ability to perform outcome measures;
    8. Significant spinal deformity and/or presence of spinal rods, which would interfere with positioning or imaging of the subject in the MRI scanner, including, but not limited to, severe scoliosis (ie, Cobb angle ≥ 30 degrees);
    9. Body mass index for age ≥ 95th percentile;
    10. For children (aged 4 years to 11 years, inclusive), loss of 30 degrees of plantar flexion from the normal range of movement at the ankle joint due to contracture;
    11. A need, in the Investigator’s opinion, to undergo an invasive procedure (eg, spinal fusion or surgical contracture release) for 2 years after randomization;
    12. Exposure to another investigational drug within 3 months prior to Screening or 5 half-lives since last administration, whichever is longer;
    13. Exposure to approved or investigational drugs that may affect dystrophin or utrophin expression in the previous 6 months prior to Screening; or
    14. Exposure to an approved or investigational gene transfer drug.
    E.5 End points
    E.5.1Primary end point(s)
    Primary safety endpoints:
     Incidence of adverse events from baseline to 12 months;
     Incidence of clinical laboratory abnormalities from baseline to 12 months;
     Incidence of abnormalities in vital signs and physical examinations from baseline to 12 months; and
     Incidence of abnormalities on electrocardiograms (ECGs) from baseline to 12 months.
     Characterization of SGT-001 kinetics, immune response, and shedding:
     Viral load count from baseline to 12 months;
     Immunological responses: B-cell (total/circulating and neutralizing anti-adeno-associated virus AAV serotype 9 [AAV9] antibody titers) and T-cell reactivity (to AAV9 and microdystrophin) from baseline to 12 months;
     Vector biodistribution in blood and muscle biopsy from baseline to 12 months; and
     AAV9 vector shedding from baseline to 12 months.
    Primary efficacy endpoint:
     Change from baseline at 12 months (post SGT-001 administration) in microdystrophin protein levels in muscle biopsies (active treatment group), as quantified by WB.
    E.5.1.1Timepoint(s) of evaluation of this end point
    12 months
    E.5.2Secondary end point(s)
     Change from baseline at 12 months (post SGT-001 administration) in microdystrophin protein levels in muscle biopsies (active treatment group), as quantified by mass spectrometry;
     Change from baseline at 12 months (post SGT-001 administration) in microdystrophin protein levels in muscle biopsies (active treatment group), as quantified by IF;
     Lower and/or upper limb skeletal muscle mass and composition as measured by MRI, including changes from baseline at 12 months in edema, fat composition, cross-sectional area and contractile area in the active treatment group compared to the control group;
     Change from baseline at 12 months in the North Star Ambulatory Assessment in the active treatment group compared to the control group (ambulatory children);
     Change from baseline at 12 months in time to run 10 meters in the active treatment group compared to the control group (ambulatory children);
     Change from baseline at 12 months in time to rise from the floor in the active treatment group compared to the control group (ambulatory children);
     Changes from baseline at 12 months in upper limb function in the active treatment group compared to the control group, as measured by the Performance of the Upper Limb functional scale;
     Changes from baseline at 12 months in the muscular strength of the upper extremities in the active treatment group compared to the control group, as measured by MyoGrip and MyoPinch myometry;
     Changes from baseline at 12 months in daily motor activity in the active treatment group compared to the control group, as measured by a wearable accelerometer (Actimyo) for continuous monitoring;
     Changes from baseline at 12 months in naturalistic motor function activity in the active treatment group compared to the control group, as measured by an ambient measurement system (Echo5D) at home;
     Changes in respiratory function from baseline at 12 months in the active treatment group compared to the control group, as measured by forced vital capacity (FVC) and forced expiratory volume in 1 second, and sniff nasal inspiratory pressure;
     Changes in cardiac function from baseline at 12 months in the active treatment group compared to the control group, as measured by echocardiography (ECHO), including, but not limited to,
    ejection fraction (EF), left ventricle end systolic volume, and myocardial peak circumferential strain;
     Change from baseline at 12 months on the 6-minute walk test in the active treatment group compared to the control group (ambulatory children);
     Changes from baseline at 12 months in exploratory serum and muscle biomarkers in the active treatment group compared to the control group;
     Changes from baseline at 12 months in quality of life and self-reported outcome measures in the active treatment group compared to the control group;
     Changes from baseline at the intermediate timepoints (Day 45, 3, 6, and 9 months) in microdystrophin in muscle biopsies, as quantified by WB, mass spectrometry, and IF (active treatment group);
     For the above endpoints, changes from baseline to intermediate timepoints (Day 45, 3, 6, and 9 months) in the active treatment group compared to the control group;
     Changes from baseline at 12 months (and intermediate timepoints) in microdystrophin in muscle biopsies, as quantified by WB, mass spectrometry, and IF (combined active treatment and delayed treatment groups; post SGT-001 treatment); and
     For the above endpoints, change from baseline at 12 months (combined active treatment and delayed treatment groups; post SGT-001 treatment).
    E.5.2.1Timepoint(s) of evaluation of this end point
    12 months
    E.6 and E.7 Scope of the trial
    E.6Scope of the trial
    E.6.1Diagnosis No
    E.6.2Prophylaxis No
    E.6.3Therapy No
    E.6.4Safety Yes
    E.6.5Efficacy Yes
    E.6.6Pharmacokinetic Yes
    E.6.7Pharmacodynamic Yes
    E.6.8Bioequivalence No
    E.6.9Dose response Yes
    E.6.10Pharmacogenetic No
    E.6.11Pharmacogenomic No
    E.6.12Pharmacoeconomic No
    E.6.13Others No
    E.7Trial type and phase
    E.7.1Human pharmacology (Phase I) Yes
    E.7.1.1First administration to humans Yes
    E.7.1.2Bioequivalence study No
    E.7.1.3Other No
    E.7.1.3.1Other trial type description
    E.7.2Therapeutic exploratory (Phase II) Yes
    E.7.3Therapeutic confirmatory (Phase III) No
    E.7.4Therapeutic use (Phase IV) No
    E.8 Design of the trial
    E.8.1Controlled Yes
    E.8.1.1Randomised Yes
    E.8.1.2Open Yes
    E.8.1.3Single blind No
    E.8.1.4Double blind No
    E.8.1.5Parallel group Yes
    E.8.1.6Cross over No
    E.8.1.7Other No
    E.8.2 Comparator of controlled trial
    E.8.2.1Other medicinal product(s) No
    E.8.2.2Placebo No
    E.8.2.3Other Yes
    E.8.2.3.1Comparator description
    Open-label untreated control
    E.8.2.4Number of treatment arms in the trial3
    E.8.3 The trial involves single site in the Member State concerned Yes
    E.8.4 The trial involves multiple sites in the Member State concerned No
    E.8.5The trial involves multiple Member States No
    E.8.6 Trial involving sites outside the EEA
    E.8.6.1Trial being conducted both within and outside the EEA Yes
    E.8.6.2Trial being conducted completely outside of the EEA No
    E.8.6.3If E.8.6.1 or E.8.6.2 are Yes, specify the regions in which trial sites are planned
    United Kingdom
    United States
    E.8.7Trial has a data monitoring committee Yes
    E.8.8 Definition of the end of the trial and justification where it is not the last visit of the last subject undergoing the trial
    LVLS
    E.8.9 Initial estimate of the duration of the trial
    E.8.9.1In the Member State concerned years7
    E.8.9.1In the Member State concerned months6
    E.8.9.1In the Member State concerned days
    E.8.9.2In all countries concerned by the trial years7
    E.8.9.2In all countries concerned by the trial months6
    F. Population of Trial Subjects
    F.1 Age Range
    F.1.1Trial has subjects under 18 Yes
    F.1.1Number of subjects for this age range: 16
    F.1.1.1In Utero No
    F.1.1.2Preterm newborn infants (up to gestational age < 37 weeks) No
    F.1.1.3Newborns (0-27 days) No
    F.1.1.4Infants and toddlers (28 days-23 months) No
    F.1.1.5Children (2-11years) Yes
    F.1.1.5.1Number of subjects for this age range: 8
    F.1.1.6Adolescents (12-17 years) Yes
    F.1.1.6.1Number of subjects for this age range: 8
    F.1.2Adults (18-64 years) No
    F.1.3Elderly (>=65 years) No
    F.2 Gender
    F.2.1Female No
    F.2.2Male Yes
    F.3 Group of trial subjects
    F.3.1Healthy volunteers No
    F.3.2Patients Yes
    F.3.3Specific vulnerable populations Yes
    F.3.3.1Women of childbearing potential not using contraception No
    F.3.3.2Women of child-bearing potential using contraception No
    F.3.3.3Pregnant women No
    F.3.3.4Nursing women No
    F.3.3.5Emergency situation No
    F.3.3.6Subjects incapable of giving consent personally Yes
    F.3.3.6.1Details of subjects incapable of giving consent
    Parent and Guardian will provide consent to children under 17 years old. Children under 17 years old will also sign an assent.
    F.3.3.7Others No
    F.4 Planned number of subjects to be included
    F.4.1In the member state8
    F.4.2 For a multinational trial
    F.4.2.1In the EEA 8
    F.4.2.2In the whole clinical trial 32
    F.5 Plans for treatment or care after the subject has ended the participation in the trial (if it is different from the expected normal treatment of that condition)
    Standard of Care
    G. Investigator Networks to be involved in the Trial
    N. Review by the Competent Authority or Ethics Committee in the country concerned
    N.Competent Authority Decision Authorised
    N.Date of Competent Authority Decision2019-01-08
    N.Ethics Committee Opinion of the trial applicationFavourable
    N.Ethics Committee Opinion: Reason(s) for unfavourable opinion
    N.Date of Ethics Committee Opinion2018-10-29
    P. End of Trial
    P.End of Trial StatusGB - no longer in EU/EEA
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